Elevidys Gene Therapy is still being approached with skepticism by countries due to its price tag of 3 million US dollars and the lack of sufficient clinical evidence of improvement. Chile was also among the countries that placed reservations. The Chilean Ministry of Health also explained why Elevidys gene therapy was not included in the reimbursement scope.
Chilean Health Minister Shares Her Thoughts on Elevidys
Chilean Health Minister Ximena Aguilera explained the non-supply of Elevidys, a drug used to treat Duchenne Muscular Dystrophy, arguing that “there is no clinical evidence of improvement”. [Read More: Clinical Trials Review of Elevidys Gene Therapy]
It is estimated that around 800 children in the country suffer from Duchenne Muscular Dystrophy and the Ministry of Health has refused funding for the Elevidys gene therapy through the Institute of Public Health due to insufficient evidence proving its effectiveness.
There is Not Enough Clinical Evidence That Elevidys Improves Patients’ Health
Chilean Health Minister Ximena Aguilera stated, “Although there are results at the molecular level, there is no clinical evidence of improvement yet.”
The Minister of Health said that for this reason, the Public Health Institute has not registered the drug and therefore “they cannot finance this drug.”
Meanwhile, Duchenne Families Association President Marcos Reyes, who attended the march for his two children who contracted the disease, said there had been no communication with the health authority and lamented the ministry’s failure to make progress on an agenda targeting the disease.
Families Continue Individual Campaigns
Faced with the fact that the Elevidys gene therapy will not be covered by the government, parents are continuing their individual campaigns to obtain resources to finance the treatment of this disorder through voluntary donations.
Sarepta Still Hasn’t Lowered Price of Elevidys Gene Therapy
While Sarepta has not yet provided convincing clinical evidence for its gene therapy, it has been met with great opposition from patients’ families because the treatment costs $3 million. [Learn More: Frequently Asked Questions About Elevidys]
Sarepta shares lost approximately 28% of their value after a 16-year-old patient who received Elevidys gene therapy died on March 18, 2025. Sarepta and Roche, who were the focus of criticism, failed to provide convincing explanations. The money-hungry company, which demanded $3 million for a one-time treatment, stated that the patient’s cause of death was Cytomegalovirus Infection. [Read More]
